Health economic assessments of anticancer agents: A comparison of assessment technologies applied to end-stage cancer.

PMU1 MEASUREMENT HEALTH OUTCOMES ASSOCIATED WITH MEDICINES AT A NATIONAL LEVEL

To perform a cost-effectiveness analysis of the use of Atorvastatin 10 mg in the primary prevention of cardiovascular disease in patients with type 2 diabetes (DM2). A deterministic and retrospective model by a decision analysis based on CARDS study (Collaborative Atorvastatin Diabetes Study) was performed. In the CARDS study, a significant reduction in cardiovascular morbimortality by the use of Atorvastatin 10 mg versus placebo (5.8 vs. 9.0%, p=0.001) in DM2 patients with an additional condition, had previously been demonstrated. In the present cost-effectiveness analysis, effectiveness units were life years gained (LYG) and quality adjusted life years (QALY), obtained from differences in morbimortality and life expectancy in DM2 patients, with and without previous cardiovascular events. Costs of the evaluated alternatives were obtained from the CARDS results. Incremental cost-effectiveness ratio of using Atorvastin 10 mg versus placebo was 5,886 euro per LYG and 8,046 euro per QALY. Sensitivity analyses confirmed the model stability. In the primary prevention of the cardiovascular disease in type 2 diabetic patients, the use of Atorvastatin 10 mg is cost-effective, with a cost per LYG and per QALY below that of other alternatives widely used in the Spanish National Health System, and also below a value considered as a reasonable threshold for our country, which might unofficialy be around 30,000 euro/ QALY.

Cost-Effectiveness of Rituximab for Maintenance in Patients with Follicular Non-Hodgkin's Lymphoma in the Dutch Setting

Evaluate costs and benefits of rituximab in combination with cyclophosphamide/vincristine/prednisolone chemotherapy regimen (R-CVP), in previously untreated patients with indolent non-Hodgkin lymphoma (NHL), compared to CVP alone from a Portuguese National Health System (NHS) perspective. Cost-effectiveness (Life Years Gained--LYG) and cost-utility analysis (Quality Adjusted Life Years--QALYs) were performed for a time horizon of 10 years, according to a Markov economic model with three health states (progression free survival, progression and death) and monthly cycles for a population of previously untreated patients with indolent NHL. Data from a phase III clinical trial was used and expanded to include unpublished 53-month median follow-up data. Survival after first-line therapy was estimated from the Scotland and Newcastle Lymphoma Group registry data and utilities were derived from a study in the UK performed in patients with follicular lymphoma. Resource consumption was estimated by a Portuguese expert panel (Delbecq Panel). Costs were calculated from the Portuguese NHS perspective through official data with prices updated to 2008. Only direct medical costs were considered. Costs and clinical outcomes were discounted at 5% per annum. Deterministic and probabilistic sensitivity analysis were performed around assumptions on the time horizon, costs, utilities and excess mortality rate due to progression applied in the base-case analysis. The 10-year base-case analysis showed a lower total cost per patient with CVP alone (€ 85,838) in comparison with R-CVP (€ 87,774). Life expectancy and Quality adjusted life expectancy per patient were higher with R-CVP (6.361 and 4.166, respectively) than with CVP alone (5.557 and 3.438, respectively), representing increases of 0.804 in LYG and 0.728 (8.7 months) in QALYs gained. The incremental cost per LYG was € 2,407 and the incremental cost per QALY gained was € 2,661. The probabilistic sensitivity analysis confirmed the robustness of the base-case analysis results. This study demonstrates that the combination R-CVP in previously untreated indolent NHL patients improves life expectancy and is a cost-effective alternative to CVP in Portugal.

Despite the important advances observed in the last 25 years in the comprehension of the clinical and biological nature of breast cancer and its treatment, this disease remains a significant cause of cancer morbidity and mortality worldwide. The clinical trial Hera has demonstrated the safety and efficacy of trastuzumab in the treatment of HER2 positive (HER2+) breast cancer patients, in early stages, subsequent to surgery, chemotherapy (neoadjuvante or adjuvant) and radiotherapy, if applied. To evaluate the cost-effectiveness of 1-year trastuzumab treatment versus standard care (observation following standard adjuvant chemotherapy), in patients with HER2+ breast cancer in early stages from the societal and the Portuguese National Health Service (NHS) perspectives. A 5-state Markov model with annual transition cycles was developed to estimate the long term health and economic outcomes of HER2+ early breast cáncer patients based on HERA clinical trial results. Portuguese NHS resource use and costs were estimated from a consensus expert panel and published unit costs, respectively. Clinical and economic outcomes were discounted at 3% per annum. The incremental cost-effectiveness ratios per life year gained (LYG) and per quality adjusted life year (QALY) gained were estimated. One-way sensitivity analysis was performed. Considering a 45 year time horizon, treatment with trastuzumab was estimated to increase discounted life expectancy by 2,114 life years and quality-adjusted life expectancy by 2,009 QALYs compared to standard care. Direct and indirect costs were projected to be 61.839 euro and 19.759 euro with trastuzumab and 40.559 euro and 25.392 euro with standard of care. These results corresponded to incremental cost-effectiveness ratios of 10.067 euro and 10.595 euro assuming direct costs only, and of 7.400 euro and 7.789 euro including indirect costs, per life year gained and per QALY gained, respectively. The 1-year trastuzumab use as adjuvant therapy in HER-2+ early breast cancer patients improves survival and can be considered a cost effective therapy with a high degree of certainty in the Portuguese setting.

5583 Background: FDA has approved three novel AAs [Apalutamide(A), Darolutamide(D) and Enzalutamide(E)] in combination with Androgen deprivation therapy ( ADT) for treatment of (nmCRPC) patients (pts). We report the cost-effectiveness of these drugs from the US perspective to help facilitate the choice of these agents for clinical practice. Methods: A life time Markov state-transition model was constructed with three health states (Metastasis-Free Survival[MFS], Metastatic disease, and Death) to compare cost-effectiveness of AA therapies for treatment of nmCRPC based on US healthcare payer perspective. A network meta-analysis of MFS and OS was conducted due to the lack of head to head trials. An approximation of the original individual-level patient time-to-event data were derived from digitized Kaplan-Meier curves for OS and MFS. Weibull distributions was selected as the best fitted model fitted and extrapolated as per the NICE decision support unit recommendations. Medication costs were based on wholesale acquisition cost. Adverse event (AE) grades 3/4 management costs were incorporated in the model. Discount rate of 3% per year was applied to costs and effects. Life years (LYs) and quality adjusted life years (QALYs) for each treatment as well as the incremental cost effectiveness (ICER) and cost utility (ICUR) ratios were estimated. Base case analyses (BCA) and probabilistic sensitivity analyses (PSA) were estimated. Results: The table summarizes the results form BCA analyses. A+ADT offers best gain in LYs (8.37yrs) and QALYs (5.30 yrs) but at higher cost. Conclusions: Apalutamide was associated with gains in LYs and QALYs traded off with higher lifetime cost relative to other AA alternatives. ADT was associated with lower gains in LYs and QALYs traded off with lower lifetime cost relative to other alternatives. Based on a $150,000/QALY threshold pay off, A+ADT is likely more cost effective compared to E+ADT or ADT alone; while E+ ADT may be more cost effective compared to D+ ADT. [Table: see text]

BackgroundSevere sepsis is a leading cause of mortality in intensive care units (ICUs). Efficient and cost effective use of antibiotics is necessary for improving treatment outcomes.PurposeThe aim was to investigate...

Ceftazidime-avibactam (C/A), has shown reduction in mortality rates and risk of nephrotoxicity, compared to colistin, conventional therapy. To estimate the cost-effectiveness of C/A versus colistin + meropenem in the treatment of infections due to carbapenem-resistant Enterobacteriaceae (CRE) in Chile. An economic decision tree type model was adapted. The perspective of the public payer was used with a time horizon of 30 days and extrapolation to life expectancy. The clinical information was derived from an observational study. Medication and care costs correspond to local reports. The results are expressed as incremental cost-effectiveness ratio (ICER) per life year gained (LYG) and per quality adjusted life year (QALY) in Chilean pesos and US dollars (US$ 1.00 = $792.2218). 8.65 and 6.48 LYGs and 6.44 and 4.27 QALYs were obtained, for C/A and colistin + meropenem, respectively. The estimated ICER for C/A was $940,488 (US$1,187.2) per AVG and $938,715 (US$1,184.9) per QALY. Given the lack of publications or evidence, the model is based on an observational study. C/A would reduce the death rate and increase LYGs and QALYs, resulting in a cost-effective alternative vs. colistin + meropenem for CRE.

Obinutuzumab and Chlorambucil Versus Chlorambucil Monotherapy for Treatment of Previously Untreated Chronic Lymphocytic Leukemia Where Fludarabine-Based Chemotherapy Is Considered Inappropriate: A Canadian Cost-Utility Analysis

A Mediterranean Diet Is Cost-Effective in Patients with Previous Myocardial Infarction1–3

6528 Background: CO.17 study is the first intergroup (Canadian/Australian) trial, that prospectively collected resource utilization and utility data for cetuximab (N=283) vs. best supportive care alone (BSC; N=274) in advanced colorectal cancer patients. A cost-effectiveness analysis was conducted based on the improved survival, higher cost of cetuximab and variable costs across countries from the perspective of the Canadian health care system. Methods: The mean difference in trial survival times between cetuximab and BSC was calculated. Direct medical resource utilization data was collected during the trial from the time of randomization until death or study closure. Trial resources included medications physician visits, toxicity management, institutionalization, emergency department visits and hospitalizations. Mean overall incremental cost (2007 $CAN-provincial sources) was determined. Drug acquisition costs (DAC) for cetuximab, based on several countries, were used to determine the incremental cost effectiveness ratio (ICER) in dollars per life-year gained (LYG). Sensitivity analyses for cetuximab DACs were conducted. Bootstrapping (1,000 iterations) provided 95%CI. Results: Mean incremental trial survival was 0.12 years. Preliminary results showed variability in the ICER, where DAC was the cost driver. At the lowest DAC ($2.94/mg), overall incremental costs were $19,361 for cetuximab compared to BSC, with incremental values of $1,086 for toxicity management, $1,016 for hospitalization, and $410 plus $81 for oncologist and family physician visits respectively. The ICER for cetuximab was $183,287/LYG (95%CI: $114,139-$581,027) using a $2.94/mg DAC (Switzerland), $198,467/LYG ($123,509-$558,270; $3.24/mg-Canadian suggested) and $375,047/LYG ($237,139- $1,233,111; $6.73/mg US). Sensitivity analyses produced an ICER of $50,000/LYG at $0.28/mg; $100,000/LYG at $1.30/mg and $200,000/LYG at $3.27/mg. Cost per quality adjusted life year will also be determined. Conclusions: Cetuximab showed high and variable ICERs dependent on a range of DACs in CO.17 advanced colorectal cancer patients. Utility will impact the incremental value. No significant financial relationships to disclose.

Although the FDA Accelerated Approval Program (AAP) has come under scrutiny, the population-level health benefit of the program has not been quantified. Therefore, the objective of this study was to estimate the number of life years gained among patients with cancer that can be attributable to the therapies receiving FDA accelerated approvals in oncology between 2006 and 2022 in the United States. The data sources used were FDA listings, FDA approval letters and labels, published clinical trial data and other publications including relative effectiveness estimates, and the Ipsos Oncology Uptake Tool for product uptake. Data for 130 oncology treatments approved by the FDA under the AAP were extracted and validated. We developed a decision analytic model to estimate the survival gain for each indication and to accumulate life years gained for consecutive cohorts of patients receiving the therapies. Life year gains were estimated with and without the AAP, and the incremental life years gained were attributed to the program. The analysis estimated that through December 2022 in the United States, the program gained approximately 263,000 life years across 69 products for which overall survival data were available, for approximately 911,000 patients with cancer. Policy discussions about the evaluation of AAP cannot be complete without assessing its impact on its most important target outcome: patient survival. To date, there has been no estimation of the life year gain delivered by the AAP. Our research shows that substantial number of life years were gained for patients with high unmet need by the cancer therapies approved through the program.

Background There are no studies that have specifically investigated the cost-effectiveness of cascade screening of children for heterozygous familial hypercholesterolemia (FH) and treatment of affected individuals with statins to prevent coronary heart disease (CHD). Purpose This study explores the cost-effectiveness of this strategy from the perspective of the Australian public healthcare system. Methods A lifetime Markov model with four health states (Alive without CHD, Alive with CHD, Dead from fatal CHD, and Dead from other causes) was developed to simulate the progression of ten- year-old children screened for FH and treated immediately with statins if found to have FH. The underlying prevalence of FH in this target population was assumed to be 56.8%, and the sensitivity and specificity of testing was 100%. The comparator was usual care, which assumed that subjects started statins spontaneously at a later point or when they experienced a cardiovascular event. The effect of reducing low-density lipoprotein cholesterol (LDL-C) on the risk of a first event at each age assumed that risk was proportional to total lifetime exposure and was implemented using Mendelian randomisation analysis data. Cost and other outcome data were sourced from published sources. Outcome of interests were costs in Australian dollars (AUD), life years gained (LYG) and quality-adjusted life years (QALYs) gained, as well as incremental cost-effectiveness ratios (ICERs) of costs per LYG and per QALY gained. All future costs and outcomes were discounted by 5% annually. Results Undiscounted results showed that compared with usual care, cascade screening of ten year-old children for FH and initiation of treatment of affected individuals saved 7.77 LYG and 7.53 QALYs per person over a lifetime. With 5% annual discounting, there were 0.97 LYG and 1.07 QALYs gained per person, at an additional cost of $3,244. These equated to ICERs of $3334 per LYG and $3023 per QALY gained. The equivalent ICERs in USD would be $5089 per LYG gained and $4615 per QALY gained. Sensitivity analysis showed the results to be robust. Conclusions Compared to usual care, cascade screening of ten year old children for FH and treating affected individuals is likely to be highly cost-effective. Table 1. Granular cost and benefit data Funding Acknowledgement Type of funding source: None

PCN102 COST-EFFECTIVENESS OF NIVOLUMAB VERSUS DACARBAZINE IN FIRST-LINE TREATMENT OF ADVANCED MELANOMA FROM BRAZILIAN PUBLIC HEALTHCARE SYSTEM PERSPECTIVE

Economic evaluation of the Mediterranean diet for patients following first AMI (acute myocardial infarction). A cost utility analysis was conducted, using a Markov model to describe health status, costs, quality of life and deaths to compare the Mediterranean diet to a prudent Western diet. Program effectiveness was based on the Lyon Diet Heart Study. Costs were estimated in $AU, (and converted to $US and €Euros) based on reported resource use. Performance was measured as cost per quality adjusted life year (QALY) gained. Extensive one-way sensitivity analyses were performed. The Mediterranean diet was estimated to cost less and be more effective (dominant) compared with a prudent Western diet. There was a mean gain in life years of 0.56 per person and a gain in quality adjusted life years of 0.61 per person. Based on the published results from the Lyon Diet Heart Study, and conservative assumptions, the Mediterranean diet is cost saving for persons following first AMI when modeled over 10 years. Policy makers and clinicians should strongly consider application of results to their own setting.